Scientific Overview

Androgen deprivation therapy remains the primary first line therapy for advanced prostate cancer but the current products, such as LUPRON® and ELIGARD® (leuprolide), FIRMAGON® (degarelix), and ZOLADEX® (goserelin) have several important shortfalls. LUPRON, ELIGARD, and ZOLADEX are LHRH agonists whose initial administration leads to an initial 14 to 21-day testosterone surge (flare) and interval micro increases (spikes or escapes) in testosterone blood levels. FIRMAGON, a GnRH antagonist, is a large-volume subcutaneous formulation designed for only a single month release. FIRMAGON requires a loading dose of two 3 mL subcutaneous injections followed by a 4 mL maintenance subcutaneous injection. The 4 mL maintenance subcutaneous injection has to be repeated monthly. In contrast, VERU-100 is designed to address a number of these important shortfalls of currently marketed ADT products: VERU-100 is a long-acting GnRH antagonist designed to be administered as a small volume (<1 mL) subcutaneous 3-month depot injection without a loading dose. VERU-100, as a GnRH antagonist, immediately suppresses testosterone with no testosterone surge upon initial or repeated administration unlike what occurs with the currently approved LHRH agonists.

Determination of the ability of a novel, long-acting subcutaneous GnRH antagonist, VERU-100, to castrate without a testosterone surge in a rat model(Click image to enlarge)

VERU-100

Development Plan

The Company had a Pre-IND meeting with the FDA in May 2019 clarifying the requirements for regulatory development pathway. FDA agreed to an expedited regulatory development pathway for VERU-100. The Company plans to conduct a single Phase 2 open label, multicenter dose finding clinical study of three doses of VERU-100 in men with advanced prostate cancer (n=35) for a single 3 month injection, and a single Phase 3- open label single arm study in men with advanced prostate cancer (n=100) as the pivotal, registration study for NDA submission. The Company plans to submit an IND in early calendar year 2020 and then initiate the Phase 2 dose finding study.

VERU-100

Market

VERU-100 is a long-acting GnRH antagonist for ADT designed to be administered as a small volume subcutaneous 3-month depot injection without a loading dose. As a GnRH antagonist, VERU-100 should immediately suppress testosterone with no testosterone surge upon initial or repeated administration and no testosterone micro-increases which may adversely affect patient outcomes1—a problem which potentially occurs with approved LHRH agonist drugs like LUPRON®, ZOLADEX® and ELIGARD®. Currently, there are no GnRH antagonists commercially approved beyond a 1-month depot injection, making VERU-100, if approved, the only commercially available GnRH antagonist 3-month depot. Total global sales of ADT drugs in 2018 were $2.6 billion.

Getzenberg, et al., “Determination of the ability of a novel, long-acting subcutaneous GnRH antagonist, VERU-100. to castrate without a testosterone surge in a rat model, GU ASCO Poster, 2020